| Gene Therapy for Retina to Prevent Recurrence of Neovascularization 
Description A new gene therapy to suppress or prevent recurrence of Neovascularization (NV) following laser therapy for choroidal neovascularization (CNV) or diabetic retinopathy. Glial fibrillary acidic protein (GFAP) is upregulated in Muller cells in retinal regions that have been lasered. Using the GFAP promoter to deliver anti-angiogenic factors has the novel feature of permitting laser-activated gene therapy that is limited to the specific retinal foci needing treatment, reducing exposure of surrounding tissues to anti-angiogenic therapy. The anti-angiogenic therapy would be more effective because it would be used to suppress neovascularization at its initiation, when the blood vessels are far more sensitive to antiangiogenic therapy. Upregulation of GFAP also occurs in focal regions of retinal degeneration. Transfection with viral constructs incorporating a GFAP promoter and a neuroprotective gene would result in an auto-initiated gene therapy to prevent, slow or delay progressive photoreceptor death in geographic atrophy in age-related macular degeneration, inherited retinal dystrophies or environmental injury. This approach would prolong useful vision in sick retinas. An advantage of auto-initiated gene therapy is that intervention would begin when the atrophic region is small, providing immediate protection to the stressed at the edges of the atrophy. Vision would be preserved by slowing or preventing cell death whereas the reduced volume of degenerating retina may avoid associated inflammatory responses and the bystander effect, i.e. photoreceptor death as a consequence of the cascade of events initiated by death of a neighboring cell. Background of Invention Neovascularization is a serious complication in both diabetic retinopathy and age-related macular degeneration, often leading to blindness. The current treatment for both is to create small laser burns in the retina. These treatments slow loss of vision, but recurrence is a common problem, leading to progressive vision loss and even blindness. Potential Applications Retinal gene therapy that is auto-initiated in pathology, or laser-activated. 
Photo credit: Hunkeler Eye Institute
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Updated August 10, 2008 | 
